The National Institute for Health and Care Excellence (NICE) have approved Akcea Therapeutics hereditary transthyretin amyloidosis (hATTR) treatment inotersen, brand name Tegsedi▼, for the treatment of polyneuropathy in adult patients with hATTR.
This means that Tegsedi can be prescribed and funded by the NHS in England and Wales.
Inotersen is recommended as an option for treating stage 1 and stage 2 polyneuropathy in adults with hereditary transthyretin amyloidosis. It is recommended only if the company provides inotersen according to the commercial arrangement.
Polyneuropathy is damage or disease affecting peripheral nerves (peripheral neuropathy) in roughly the same areas on both sides of the body, featuring weakness, numbness, and burning pain.
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Why the NICE made these recommendations
Hereditary transthyretin amyloidosis is a rare condition that severely affects the quality of life of people with the condition, and their families and carers. Current treatment is supportive care.
Clinical trial evidence shows that inotersen slows progression of the disease considerably, although its long-term benefits are uncertain. Despite the uncertainties, inotersen is likely to provide important clinical benefits for people with hereditary transthyretin amyloidosis and value for money within the context of a highly specialised service. It is therefore recommended for use in the NHS.
About Tegsedi source EMA)
Tegsedi is a medicine used to treat nerve damage caused by hereditary transthyretin amyloidosis (hATTR), a disease in which proteins called amyloids build up in tissues around the body including around the nerves.
Tegsedi is used in adult patients in the first two stages of the nerve damage (stage 1, when the patient is able to walk unaided, and stage 2, when the patient can still walk but needs help).
hATTR is rare, and Tegsedi was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 26 March 2014.
In patients with hATTR, a protein called transthyretin which circulates in the blood is defective and breaks easily. The broken protein forms amyloid deposits in tissues and organs around the body, including around nerves, where it interferes with their normal functions.
The active substance in Tegsedi, inotersen, is an ‘antisense oligonucleotide’, a very short piece of synthetic genetic material that has been designed to attach to and block the genetic material of the cell responsible for producing transthyretin. This reduces production of transthyretin, thereby reducing the formation of amyloids and relieving the symptoms of hATTR amyloidosis.
Tegsedi was shown to be effective in the treatment of stage 1 or stage 2 nerve damage in patients with hATTR; available data were not sufficient to assume a beneficial effect in stage 3 patients (those confined to a wheelchair). In light of the unmet medical need, the safety profile of Tegsedi was considered acceptable and the risks to be manageable with specific monitoring, dose reduction and stopping rules.
The European Medicines Agency therefore decided that Tegsedi’s benefits are greater than its risks and it can be authorised for use in the EU.
Tegsedi was first made available in the EU in 2018. It is manufactured by Akcea Therapeutics .
European Medicines Agency website page for Tegsedi
Links available in full article
© NICE 
Inotersen for treating hereditary transthyretin amyloidosis. Highly specialised technologies guidance [HST9] Published date: 22 May 2019
Available from: See Link below. All rights reserved. Subject to Notice of rights
NICE guidance is prepared for the National Health Service in England. All NICE guidance is subject to regular review and may be updated or withdrawn. NICE accepts no responsibility for the use of its content in this product/publication.
The information provided by NICE was accurate at the time this article was issued.
NICE information on inotersen (Tegsedi▼)
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